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Target intelligence / Profile preview
CEP290 pre-mRNA
- Target
- CEP290 pre-mRNA
- Molecular classification
- RNA, pre-mRNA
01
Overview
CEP290 pre-mRNA is the precursor messenger RNA transcribed from the CEP290 gene. It contains both exons and introns and is the target of splicing regulation. Aberrant splicing of CEP290 pre-mRNA, often due to mutations, leads to diseases like Leber Congenital Amaurosis. Therapies such as antisense oligonucleotides target this pre-mRNA to correct splicing defects.
- Other names
- CEP290 precursor mRNACEP290 primary transcript
02
Mechanism of action
Modulation of pre-mRNA splicing to restore normal mRNA production.
03
Biological functions
mRNA processingSplicing regulationGene expressionCiliogenesis (indirectly via mature mRNA)
04
Disease associations
Leber Congenital Amaurosis (LCA)Ciliopathies (indirectly via mature mRNA)
05
Safety considerations
Off-target splicing effectsImmune response to oligonucleotides or gene editing toolsLong-term efficacy and safety of splicing modulation
06
Interacting drugs
Sepofarsen
07
Biomarkers
CEP290 pre-mRNA splicing patternsPresence of specific CEP290 pre-mRNA mutations (e.g., IVS26 c.2991+1655A>G)
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